There is currently no treatment available for any aspect of CLN5 disease and there is an overwhelming need to develop a treatment of this lethal condition.
Gene therapy is a relatively new treatment technology that holds immense potential for treating patients with CLN5 disease. The University of Rochester Medical Center (URMC) will serve as the lead study site in the U.S. for an experimental treatment being developed by Neurogene for CLN5 Batten disease. This trial will move research forward in developing a potentially disease-modifying treatment for CLN5 disease, providing hope to individuals and families where currently none exists.
CLN5